Clinical trials: Bone Marrow Failure Syndromes, open to volunteers
Aplastic Anemias
Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study
Aplastic anemia (AA) is a rare bone marrow failure syndrome with an annual incidence of about 0.74/100,000, affecting all ages but more common in the elderly.
- Trial name
- NCT06650553
- Testing
- Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group.
- Where
- China
- Volunteers
- 110 people
- Sponsor
- Shanxi Bethune Hospital
Fanconi Anemia, Severe Aplastic Anemia
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine…
- Conditions
- Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes, T Cell Receptor Alpha/Beta Depletion
- Trial name
- NCT03579875
- Testing
- Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU)
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Aplastic Anaemia
HID-HSCT Versus IST as First-line Treatment for SAA
This study aims to compare the efficacy and safety of HLA-haploidentical hematopoietic stem cell transplantation (HLA-haplo HSCT) versus optimal immunosuppressive therapy (IST) as first-line treatments for severe aplastic anemia…
- Trial name
- NCT07338422
- Testing
- IST (ATG + CsA+TPORA), tranplantation
- Volunteers
- 116 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Acute Leukemia, Adenomatous Polyposis
Familial Investigations of Childhood Cancer Predisposition
NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll.
- Conditions
- Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML
- Trial name
- SJFAMILY (NCT03050268)
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- St. Jude Children's Research Hospital
Fanconi Anemia, Head and Neck Squamous Cell Carcinoma
Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC)
This research study is a phase Ib/II, single-arm, non-randomized, non-blind, multicenter study designed to determine whether Afatinib is effective and safe in patients with locoregionally unresectable and / or metastatic HNSCC…
- Trial name
- AFAN (NCT06648096)
- Testing
- Afatinib
- Where
- 2 sites in Germany, Spain
- Volunteers
- 25 people
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Background: Fanconi anemia (FA) is an inherited disorder. People with FA are more likely to get certain cancers, especially squamous cell carcinoma (SCC).
- Trial name
- NCT05687149
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- National Cancer Institute (NCI)
AML, ALL
UAB 2419-CD34 Selection Using the Automated CliniMACS Prodigy
Patients with graft failure or delayed engraftment may benefit from a hematopoietic stem cell boost or an additional hematopoietic stem cell transplantation procedure.
- Conditions
- AML, ALL, Lymphoid Malignancies, Myelodysplastic Syndromes
- Trial name
- NCT06047886
- Testing
- Infusion of CD34 selected hematopoietic stem cells
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Alabama at Birmingham
Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Refractory
Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of SAA
Efficacy and Safety of Sequential Infusion of Hypoxic 3D-Cultured Umbilical Cord Mesenchymal Stem Cells in Haploidentical Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia: A Multicenter, Randomized, Phase 1 Trial
- Conditions
- Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Refractory, Severe Aplastic Anemia
- Trial name
- NCT07299123
- Testing
- 3D hypoxia-preconditioned UC-MSC group, 2D UC-MSC group
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Chinese PLA General Hospital
Aplastic Anaemia, HSCT
Reduced-dose Conditioning Regimen Containing TBI in HSCT Treating Elderly Patients With Aplastic Anemia
The TBI-containing reduced-dose conditioning regimen was used to treat elderly patients with aplastic anemia who received hematopoietic stem cell transplantation.
- Conditions
- Aplastic Anaemia, HSCT, TBI, Old Age
- Trial name
- NCT06769568
- Testing
- reduced-dose conditioning regimen containing TBI in hematopoietic stem cell transplantation treating elderly patients with aplastic anemia
- Volunteers
- 20 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia
MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.
A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal…
- Conditions
- Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT06412497
- Testing
- Rituximab, Rabbit ATG, Cyclophosphamide
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
AML, ALL
Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic…
- Conditions
- AML, ALL, MDS, MPD Withou Myelofibrosis
- Trial name
- NCT04644016
- Testing
- Clofarabine, Fludarabine, Busulfan
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Aplastic Anemia
CK0801 for Treatment of Aplastic Anemia
This Phase 2, multicenter, open-label study is evaluating CK0801 for the treatment of aplastic anemia in adults with persistent transfusion dependence after at least one prior line of therapy or intolerance to standard-of-care…
- Trial name
- NCT07499102
- Testing
- CK0801
- Volunteers
- 12 people
- Sponsor
- Cellenkos, Inc.
Fanconi Anemia, Severe Marrow Failure
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA).
- Conditions
- Fanconi Anemia, Severe Marrow Failure, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
- Trial name
- RAFA (NCT02143830)
- Testing
- Busulfan, Cyclophosphamide, Fludarabine
- Where
- 3 sites in United States
- Volunteers
- 70 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Aplastic Anemia, Reproductive Health
Reproductive and Mental Health of Patients With Aplastic Anemia
Aplastic anemia (AA) is a hematological disease characterized by bone marrow failure, leading to varying degrees of anemia, leukopenia, and thrombocytopenia.
- Trial name
- NCT06936930
- Testing
- Sex hormone monitoring
- Where
- China
- Volunteers
- 600 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Shwachman-Diamond Syndrome, Shwachman-Diamond Syndrome-Like
Shwachman Diamond Syndrome Registry and Study
Shwachman-Diamond syndrome (SDS) is a genetic condition characterized by bone marrow failure, medical co-morbidities, and leukemia predisposition.
- Trial name
- SDSR (NCT06056908)
- Where
- 4 sites in United States
- Volunteers
- 5,000 people
- Sponsor
- Boston Children's Hospital
MDS (Myelodysplastic Syndrome), Aplastic Anemia (AA)
Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA
This study is for adults aged 18-65 with myelodysplastic syndrome (MDS) or severe aplastic anemia (AA) who are scheduled to receive a donor stem cell transplant (allogeneic hematopoietic stem cell transplant).
- Trial name
- PROMPT (NCT07400341)
- Testing
- Romiplostim, Recombinant Human Thrombopoietin
- Where
- China
- Volunteers
- 66 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Inherited Bone Marrow Failure Syndrome, Fanconi Anemia
Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data
A non-profit, translational, prospective and retrospective study. Patients will be enrolled according to the procedures described in the protocol, and samples will be processed and analyzed as specified in the protocol.
- Conditions
- Inherited Bone Marrow Failure Syndrome, Fanconi Anemia, Dyskeratosis Congenita, Telomere Biology Disorder
- Trial name
- 2023-12377081 (NCT07877727)
- Where
- 4 sites in Italy
- Volunteers
- 250 people
- Sponsor
- Istituto Giannina Gaslini
Severe Aplastic Anemia, Hematopoietic Cell Transplant
Thiotepa-Based Conditioning for Haploidentical Hematopoietic Stem Cell Transplantation in Older Patients With Severe Aplastic Anemia
This prospective, single-center observational cohort study will follow participants aged 50 years or older with severe or very severe aplastic anemia who receive haploidentical hematopoietic stem cell transplantation with a…
- Trial name
- NCT07819812
- Testing
- Thiotepa
- Volunteers
- 20 people
- Sponsor
- Peking University People's Hospital
Asherman Syndrome, Atrophic Endometrium
Bone Marrow Derived Stem Cells Mobilization for Treatment of Abnormal Endometrium
This study will assess the use of autologous bone marrow stem cells mobilization using 1,1'-[1,4-phenylenebis-(methylene)]-bis-1,4,8,11-tetraazacyclotetradecane (PLERIXAFOR) as an effective medical therapy for the treatment of…
- Conditions
- Asherman Syndrome, Atrophic Endometrium, Recurrent Implantation Failure
- Trial name
- NCT05343572
- Testing
- Plerixafor
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Hugh Taylor
Aplastic Anemia Idiopathic
Outcomes in Bone Marrow Aplasia.
Bone marrow aplasia, also known as aplastic anemia (AA) is a potentially fatal bone marrow failure syndrome characterized by a paucity of hematopoietic stem cells (HSCs) and progenitor cells with varying degrees of cytopenia and…
- Trial name
- NCT06493981
- Testing
- Eltrombopag
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- Assiut University